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Sobetirome (3,5-dimethyl-4[(4'-hydroxy-3'-isopropylbenzyl)-phenoxy] acetic acid, also known as GC-1 and QRX-431, is a member of a class of compounds known as selective thyromimetics.It was firstly developed by Thomas Scanlan’s group at the University of California-San Francisco (UCSF) in 1995 1.

In cholesterol-fed rats, sobetirome was shown to lower plasma cholesterol in a dose-dependent manner by up to 75% of untreated controls. In hypercholesterolemic mice, sobetirome was failed to induce LDL receptor mRNA expression. In different mouse models, sobetirome and T-0681 were shown to promote bile acid production and biliary sterol secretion. In cynomolgus monkeys, sobetirome was shown to reduce plasma cholesterol in a dose-dependent manner by up to 30%. Both sobetirome and T-0681 were shown to increase hepatic expression of the HDL receptor (scavenger receptor-BI, SR-BI) in animals.

In Phase I clinical trials, Sobetirome was shown to be generally well tolerated at all doses studied and proof of the therapeutic concept of cholesterol lowering was clearly demonstrated 1.

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